gene therapy, Red blood cell

UK health system to offer cutting-edge gene therapy for sickle cell disease
Britain's National Health Service (NHS) will provide a cutting-edge gene therapy that aims to cure sickle cell disease, the National Institute for Health and Care Excellence (NICE) said on Friday. The therapy from Vertex Pharmaceuticals and CRISPR Therapeutics will cost the state-funded healthcare system around 1.
Groundbreaking one-off gene therapy approved for severe sickle cell disease
People in England to benefit from groundbreaking one-off gene therapy for severe sickle cell disease approved by NICE
NICE approves Vertex’s Casgevy one-time gene therapy for sickle cell disease
The National Institute for Health and Care Excellence (NICE) has approved Vertex Pharmaceuticals’ Casgevy (exagamglogene autotemcel) gene therapy to treat a severe form of sickle cell disease (SCD).
Revolutionary gene-editing therapy for sickle cell ‘offers hope of a cure’ for NHS patients
NHS patients with sickle cell disease will be able to benefit from a groundbreaking gene-editing treatment that offers the prospect of a cure for the condition. The one-off gene therapy, known as exagamglogene autotemcel (or ‘exa-cel’),
Vertex In CASGEVY Reimbursement Deal With NHS England For Sickle Cell Disease Patients
Vertex Pharmaceuticals Inc. (VRTX), a commercial-stage global biotechnology company, announced Friday a reimbursement agreement with
Vertex wins reimbursement deal for CRISPR-partnered sickle cell therapy in England
Vertex Pharmaceuticals (VRTX) stock gains as England opts to reimburse its CRSPR (CRSP)-partnered gene therapy Casgevy to treat sickle cell disease. Read more here.
Vertex Announces CASGEVY® Reimbursement Agreement for the Treatment of Sickle Cell Disease in England
Vertex Pharmaceuticals (Nasdaq: VRTX) announced today a reimbursement agreement with NHS England for eligible sickle cell disease (SCD) patients to access the CRISPR/Cas9 gene-edited therapy, CASGEVY ® (exagamglogene autotemcel).
‘Much-needed hope’ as CRISPR gene editing therapy recommended for sickle cell disease
Experts hailed exciting news for some people with sickle cell disease in England as the health watchdog decided to grant ...
£1.65m gene-editing therapy offers hope of cure for some blood disorder patients
A £1.65 million treatment has been approved for use for some NHS patients, offering some with an inherited blood disorder ...
‘Ground-breaking’ gene treatment for sickle cell disease to be rolled out on NHS
A GROUNDBREAKING genetic treatment with the potential to cure sickle cell disease will be rolled out on the NHS. The one-off ...
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New Base Editing Gene Therapy Cures its First Patient: A Sickle-Cell Sufferer Now in the ‘Gym Every Day’
Base editing utilizes enzymes to modify single amino acids at the most foundational unit of DNA, called a base.
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'Will it really work?': Young sickle cell patient among the first to start new gene therapy
Since two groundbreaking treatments were approved in late 2023, only a handful of patients have been able to access the ...
Genetic Swiss Army Knife: New Tool For Gene Editing And Therapy
This new tool, known as minimal, versatile genetic perturbation technology (mvGPT), combines the powers of gene editing, ...
Gene Therapy Market Size is Expected to Reach USD 29.24 Billion by 2033, Growing at a CAGR of 18.60%: Straits Research
The global gene therapy market size was valued at USD 6.30 billion in 2024 and is projected to reach from USD 7.47 billion in ...
CRISPR Therapeutics to Present at the Guggenheim SMID Cap Biotech Conference
A live webcast of the fireside chat will be available on the "Events & Presentations" page in the Investors section of the Company's website at https://crisprtx.gcs-web.com/events. A replay of the ...